Tuesday, March 3, 2009

FDA rejects Genzyme’s Lumizyme

By Mass High Tech staff

Cambridge biotech giant Genzyme Corp. announced that the U.S. Food and Drug Administration has rejected the company’s initial application to market Lumizyme as a treatment for Pompe disease. Genzyme’s stock fell just under 8 percent, as of 1:30 p.m. today.

Lumizyme is the same drug as its already approved drug Myozyme, simply re-branded as it is now made in larger batches at a time.

The FDA specified the need for an agreed-upon post-approval verification study and a finalized Risk Evaluation and Mitigation Strategy. Further issues the FDA pointed out relate to concerns over the company’s Allston Landing manufacturing facility and its process controls, production equipment maintenance and microbiological monitoring and controls. The issues were noted in the fall of 2008, with Genzyme (Nasdaq: GENZ) correcting the issues last month and notifying the FDA of planned completion of the corrective measures by the end of this month. As a result, Genzyme officials called the FDA warning letter “unexpected.”

The FDA requires a “resolution” to its observations of the facility before the agency will approve Lumizyme.

In the meantime, Genzyme’s solution for treating adults with Pompe disease, an inherited disease that’s progressively debilitating and frequently fatal, is the formation of the Myozyme Temporary Access Program (MTAP) in May 2007. MTAP enables about 170 patients to receive Myozyme free under the program. Myozyme is currently only approved for the treatment of infants and children afflicted with Pompe disease, primarily because the smaller production runs Genzyme uses limits supply. Boosting the size of the production vessels would allow for the drug to be used in adults as well, under the re-branding as Lumizyme.

Pompe disease affects fewer than 10,000 people worldwide.

The company announced last week that its U.S.-approved drug for Pompe disease, Myozyme, received European approval for manufacturing the drug at the 4,000-liter bioreactor scale at the company’s Geel, Belgium-based factory.

Genzyme has more than 11,000 employees worldwide and had 2008 revenues of $4.6 billion.